by Madaline Spencer and James Radke, PhD | Mar 11, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the challenges and unmet needs of female patients with Fabry disease. Fabry disease is a type of lysosomal storage disease characterized by deficient alpha-galactosidase...
by Madaline Spencer | Mar 10, 2026
The US Food and Drug Administration (FDA) has approved Tecvayli (teclistamab-cqyv) plus Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adults with relapsed or refractory multiple myeloma (R/R MM) who have received at least one prior line of...
by Madaline Spencer | Mar 9, 2026
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp for the treatment of patients with Friedreich’s ataxia. Friedreich’s ataxia is a progressive, inherited condition that affects the nervous system and causes...
by Madaline Spencer and Scott Harwood | Mar 6, 2026
John Taggart, Head of Communications at Niemann-Pick UK (NPUK), discusses the ASMD Perspective Index and its use in diagnosing Niemann-Pick disease. Niemann-Pick disease is an inherited condition characterized by abnormal lipid metabolism that causes harmful...
by Madaline Spencer and Scott Harwood | Mar 5, 2026
Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). Mr. Stevens has been involved with the UK MPS...
